Shire Human Genetic Therapies, Inc.

Transkaryotic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Aventis Pharmaceuticals Inc.

Marianne Borowski

Douglas A Treco

Carol M Kinoshita

Richard F Selden

Kenneth Loveday

Melanie D Williams

Frances P Gillispie

Mishra Prashsant

Peter Francis Daniel

Prashant Mishra

Thomas J Schuetz

Frances P Gillespie

Peter Francis-Daniel

Carol A Kinoshita

The invention provides methods of treating &#x3b1;-galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human &#x3b1;-galactosidase A are also included.

Treatment of &#x3b1;-galactosidase a deficiency

Treatment of alpha-galactosidase a deficiency

The invention features a method of producing a high mannose glucocerebrosidase (hmGCB) which includes: providing a cell which is capable of expressing glucocerebrosidase (GCB), and allowing production of GCB having a precursor oligosaccharide under conditions which prevent the removal of at least one mannose residue distal to the pentasaccharide core of the precursor oligosaccharide of GCB, to thereby produce an hmGCB preparation. Preferably, the condition which prevents the removal of at least one mannose residue distal to the pentasaccharide core is inhibition of a class 1 processing mannosidase and/or a class 2 processing mannosidase. The invention also features an hmGCB preparation and methods of using an hmGCB preparation.

High mannose proteins and methods of making high mannose proteins

Treatment of &#x3b1;-galactosidase A deficiency

DNA molecules comprising a sequence encoding the signal peptide of hGH linked to a heterologous protein and related cells, methods and fusion proteins.

Nucleic acid encoding a chimeric polypeptide

A therapeutic method whereby an individual suspected of having an &agr;-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human &agr;-gal A, or (2) purified human &agr;-gal A obtained from cultured, genetically modified human cells.

The invention provides highly purified &agr;-Gal A, and various methods for purifying it; &agr;-Gal A preparations with altered charge and methods for making those preparations; &agr;-Gal A preparations that have an extended circulating half-life in a mammalian host, and methods for making same; and methods and dosages for administering an &agr;-Gal A preparation to a subject.

Treatment of a &agr;-galactosidase a deficiency

A therepeutic method whereby an individual suspected of having an &agr;-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human &agr;-gal A, or (2) purified human &agr;-gal A obtained from cultured, genetically modified human cells.

Therapy for &agr;-galactosidase a deficiency

A therepeutic method whereby an individual suspected of having an .alpha.-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human .alpha.-gal A, or (2) purified human .alpha.-gal A obtained from cultured, genetically modified human cells.

Growing community of inventors

Winthrop, MA, United States of America

Marianne Borowski